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Paediatrics Articles

Repurposing DFMO: Treating Rare Genetic Disorders in Children

Repurposing DFMO: A Promising Avenue for Treating Rare Genetic Disorders in Children A decades-old medication, difluoromethylornithine (DFMO or eflornithine), traditionally used for treating West African sleeping sickness, reducing unwanted facial hair in women, and preventing neuroblastoma recurrence, is now showing potential in addressing Bachmann-Bupp syndrome (BABS), an ultra-rare life-threatening genetic disorder. Affecting only about…

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